Kalydeco: New Drug Helps Some with Cystic Fibrosis

On January 31, the FDA approved ivacaftor (Kalydeco), the first-ever drug that treats an underlying cause of cystic fibrosis. In about 4% of cystic fibrosis patients, defective chloride ion channel transporters (CFTRs) are trafficked to the cell membrane, but don't work properly. Kalydeco "unlocks" and improves function of CFTR, improving lung function and quality of life. The benefits were established in 2 randomized trials of 231 patients total, in which those taking Kalydeco had a mean absolute improvement in FEV1 of ~11% predicted, sustained through one year.

About 1,200 people living with cystic fibrosis in the U.S. have the particular mutation (G551D in the CFTR gene) that would allow them to benefit from ivacaftor.

Most people with CF have the delta 508 mutation, in which defective CFTR ion channels never make it to the cell membrane. Kalydeco would not be expected to help them, but the drug is being tested in combination with other agents in clinical trials among this much larger population.

Vertex Pharmaceuticals produced Kalydeco (a.k.a. VX-770) assisted by $75 million in research funding from the Cystic Fibrosis Foundation, the leading CF patient advocacy organization. The Cystic Fibrosis Foundation will share in royalties from the sale of Kalydeco: a novel financial relationship in the highly interconnected world of industry, academia, and patient advocacy organizations. The CF Foundation told media it would reinvest its royalties into further cystic fibrosis research.

How much will ivacaftor/Kalydeco cost?

$294,000 a year (per patient)

Sales are expected to be in the hundreds of millions of dollars per year. Vertex saw their stock rise 6% on the news of FDA approval, to $37.

Back of the envelope calculations give us: 300 patients treated for a year more than pays back the $75M from Cystic Fibrosis Foundation. 1,200 patients treated for a year gives us $352,800,000…so: How much did this drug cost, in total, to develop? How much was the CEO of Vertex paid this year, in cash and stock options? How much is this going to cost Medicare Part D? I’d write to Vertex with my specific questions but I’m concerned this might put my personal safety at risk.

If I am correct, I believe the cystic fibrosis foundation is also a major stock holder in Vertex. I hope the cf foundation remembers it was started by parents to save the lives of their children. Is this foundation becoming a “big business”, I hope for our sakes it’s not.

Number needed to treat and at what cost? It is foreseeable that drug advocates will say that the costs are obviously worth it but I also imagine that the ones supporting this contention are the ones that will be reaping the benefits of such pricing whether directly or indirectly.

The hope that this drug has given people for CF is what we need to hang onto. If this drug is eventually affordable for people to get, then nothing else matters but the people who it is going to help. As a CF sufferer who was born in the 70’s, I did not think that this would be possible in my life time. I am on the drug and it has saved my life, no one could ask for a better gift. Remain positive, remain hopeful and remember the important thing is that this will save many lives. This drug company is also on the verge of treatment for everyone with CF and will continue to develop this drug so it is available for all sufferers.